This week on The Genetics Podcast, Patrick is joined by Dr. Jeffrey Chamberlain, Professor at the University of Washington School of Medicine, co-founder of Kinea Bio, and Director of the Muscular Dystrophy Cooperative Research Center of Seattle. They discuss the early breakthroughs that revealed the structure and function of the dystrophin gene, how those insights led to the creation of micro-dystrophin and systemic AAV delivery in Duchenne, and the major scientific and clinical challenges the field must now solve.

Show Notes: 

0:00 Intro to The Genetics Podcast

00:59 Welcome to Jeffrey

01:33 Early discovery of the dystrophin gene and how it shaped Duchenne muscular dystrophy (DMD) research

09:07 Efforts to map dystrophin and develop practical diagnostic techniques

12:04 How research in Jeffrey’s lab gradually led to the creation of micro-dystrophin 

20:15 How micro-dystrophin and AAV delivery converged into a viable systemic gene therapy strategy

27:23 Current successes and safety challenges in systemic AAV gene therapy for neuromuscular disease

34:44 Prospects and limitations of gene editing for Duchenne and emerging alternatives to AAV micro-dystrophin

44:57 Closing remarks

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